To those who say "impossible, impractical, unrealistic," we say:

CHALLENGE ACCEPTED

We're relentless in our pursuit of new treatments because patients shouldn't have to wait for hope.

Rose, living with acute hepatic porphyria

We are developing a new class of innovative medicines called

RNAi THERAPEUTICS

that we believe have the potential to transform the lives of people living with rare diseases.

Dane, living with hATTR amyloidosis

We are focused on diseases for which treatment options are limited or simply don't exist.

OUR ROBUST PIPELINE

includes investigational RNAi therapeutics for rare genetic, cardio-metabolic, hepatic infectious, and CNS and ocular diseases.

We’re proud to be named a

TOP PLACE TO WORK

for the 4th year in a row!

 

 

NOW APPROVED IN THE U.S.

To those who say "impossible, impractical, unrealistic,"  we say:

CHALLENGE ACCEPTED

We're relentless in our pursuit of new treatments because patients shouldn't have to wait for hope.

Rose, living with acute hepatic porphyria

We are developing a new class of innovative medicines called

RNAi THERAPEUTICS

that we believe have the potential to transform the lives of people living with rare diseases.

Dane, living with hATTR amyloidosis

We are focused on diseases for which treatment options are limited or simply don't exist.

OUR ROBUST PIPELINE

includes investigational RNAi therapeutics for rare genetic, cardio-metabolic, hepatic infectious, and CNS and ocular diseases.

LATEST NEWS

>1k
Employees
16
Countries
9
Programs Currently in Clinical Development
1
Approved Product*
>35
Clinical Studies to Date
>5yrs
Longest Duration of Exposure

Updated as of January 2019
* In the U.S. and European Union

 
 
 
 
>1k
 
 
Employees
 
 
 
 
16
 
 
Countries
 
 
 
 
9
 
 
Programs Currently in
Clinical Development
 
 
 
 
1
 
 
Approved Product*
 
 
 
 
>35
 
 
Clinical Studies
to Date
 
 
 
 
>5yrs
 
 
Longest Duration
of Exposure

Updated as of January 2019
* In the U.S. and European Union

OUR FOCUS ON PATIENTS

A Family Living with
 hATTR Amyloidosis

Candace: Living with
Porphyria

A Family Living with
Primary Hyperoxaluria Type 1 (PH1)

OUR

PIPELINE

Late Stage

Learn more about our investigational therapies in hereditary ATTR amyloidosis, hemophilia, acute hepatic porphyria, and primary hyperoxaluria type 1.

Early Stage

 

Learn more about our investigational therapies across multiple disease areas.

WORK WITH US

Join Us

We are looking for innovators to join our team—help us realize our commitment to developing a new class of medicines for patients with limited or inadequate options.

View Job Openings >

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